Efficacy Study of the Use of Sequential DFP-DFO Versus DFP (SEQDFPDFO)
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Purpose
Changes in chelation treatment and transfusion practices, during the past two decades, have dramatically improved the prognosis of thalassemia major patients.Deferiprone (DFP) has been compared with deferoxamine (DFO), using different schedules of treatment, in the majority of the 13 clinical trials published between 1990 and 2008.No statistically significant difference was shown between these two interventions during, at most, 18 months of treatment.Three randomised trials that compared sequential DFP-DFO treatment versus DFO alone reported controversial results but this could be due to small sample sizes and short treatment duration. In fact, no trial with treatment duration longer than 18 months15, which reported on mortality, adverse events, serum ferritin concentrations, as well as costs has so far been published.
This long-term sequential DFP-DFO treatment versus DFP alone treatment trial was conducted to assess the impacts of these chelation treatments on serum ferritin concentrations, mortality, adverse events, and costs in thalassemia major patients.
| Condition | Intervention | Phase |
|---|---|---|
|
Beta-Thalassemia Thalassemia Major |
Drug: Deferiprone (DFP) and Deferoxamine (DFO) Drug: Deferiprone (DFP) |
Phase 4 |
| Study Type: | Interventional |
| Study Design: | Allocation: Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Parallel Assignment Masking: Open Label Primary Purpose: Treatment |
| Official Title: | Phase IV Study of the Use of Sequential DFP-DFO Versus DFP in Thalassemia Major Patients |
- difference between multiple observations of the serum ferritin concentrations [ Time Frame: five-year treatment ] [ Designated as safety issue: Yes ]
- the difference between the T2* signal at magnetic resonance imaging (MRI) of heart and liver at T0 and T1 time (see below); survival analysis; adverse events; treatment failures; and costs. [ Time Frame: five years ] [ Designated as safety issue: Yes ]
| Enrollment: | 213 |
| Study Start Date: | September 2000 |
| Study Completion Date: | January 2008 |
| Primary Completion Date: | January 2008 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
|
Experimental: 1
Sequential treatment including DFP at 75 mg/kg, divided into three oral daily doses, for four days per week and DFO by subcutaneous infusions (8-12h) at 50 mg/kg/day for the remaining three days per week
|
Drug: Deferiprone (DFP) and Deferoxamine (DFO)
Sequential treatment including DFP at 75 mg/kg for four days per week and DFO by subcutaneous infusions (8-12h) at 50mg/kg/day for the remaining three days per week
Other Names:
|
|
Active Comparator: 2
Deferiprone alone at 75 mg/kg divided into three oral daily doses
|
Drug: Deferiprone (DFP)
DFP alone at 75 mg/kg divided into three oral daily doses
Other Name: DFP (Apotex, Canada)
|
Detailed Description:
The trial was designed as a multicentre randomised open-label trial with blinded data management and data analyses, to assess whether either treatment was superior to the other. The trial was performed on behalf of the Italian Society for the study of Thalassemia and Haemoglobinopathies (SoSTE). The investigators initiated, carried out, and controlled the trial, which was conducted without influence of the non-commercial sponsor.16
Eligibility| Ages Eligible for Study: | 13 Years to 60 Years |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Thalassemia major patients with serum ferritin concentration between 800 to 3,000 ng/ml and were over 13 years of age
Exclusion Criteria:
- Known intolerance to one of the trial treatments
- Platelet count < 100,000/mm3 or or leukocyte count < 3,000/mm3
- Severe liver damage indicated by ascites
- Heart failure
Contacts and Locations More Information
No publications provided by Azienda Ospedaliera V. Cervello
Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
| Responsible Party: | Aurelio Maggio, AO V Cervello |
| ClinicalTrials.gov Identifier: | NCT00733811 History of Changes |
| Other Study ID Numbers: | AOVCervello |
| Study First Received: | August 11, 2008 |
| Last Updated: | August 12, 2008 |
| Health Authority: | Italy: Ministry of Health |
Keywords provided by Azienda Ospedaliera V. Cervello:
|
thalassemia major chelation treatment secondary hemochromatosis |
Additional relevant MeSH terms:
|
Beta-Thalassemia Thalassemia Anemia, Hemolytic, Congenital Anemia, Hemolytic Anemia Hematologic Diseases Hemoglobinopathies Genetic Diseases, Inborn Deferoxamine Deferiprone Isoflurophate |
Siderophores Iron Chelating Agents Chelating Agents Molecular Mechanisms of Pharmacological Action Pharmacologic Actions Protease Inhibitors Enzyme Inhibitors Cholinesterase Inhibitors Cholinergic Agents Neurotransmitter Agents Physiological Effects of Drugs |
ClinicalTrials.gov processed this record on June 18, 2013